Evaluating the Psychometric Properties of Three Clinical Trial Outcome
Measures for Rett Syndrome: The purpose of this research study is to document
the psychometric properties of three non-invasive, clinic-friendly, and
quantifiable outcome measures reflecting pathologically- and clinically-
relevant symptoms of Rett Syndrome (RTT). Despite several ongoing or pending
clinical trials for pharmaceutical interventions in RTT, there are currently
no agreed-upon outcome measures sufficiently validated for use in the RTT
patient population. We have identified three potential non-invasive measures
that reflect clinical features of RTT and that have preliminary evidence of
feasibility. The next step is to document the reliability and validity of
these measures in clinical populations. We also plan to pilot an ambulatory
measure of skin temperature that has been used in other clinical populations,
as a means to evaluate peripheral temperature fluctuations and diurnal
patterns.
The study consists of two components: a virtual research visit with our
research team via Zoom and two, in-home collection periods using wearable
technology. The virtual research visit will last around 1 hour, and we will
ask parents/guardians of participants questions about their child's health,
behavior, and mood. After the virtual visit, we will also send additional
questionnaires via email for families to complete on their own time.
The two, in-home collection periods will last for 14 days, 4-6 weeks apart.
Research staff will send a sleep watch, and 3 temperature sensors to the
family's home. During these collection periods, we will ask families to have
their child wear a sleep watch (looks like a Fitbit) for 14 days. This sleep
watch will record the participant's activity when they are asleep and awake.
We will ask families to fill out a sleep diary for their child during this
time too. Families will repeat this process 4-6 weeks later.
In addition to the sleep watch, we will ask participants to wear 3, small
temperature sensors on their feet and abdomen. These temperature sensors will
be taped the participant's feet and abdomen using hypoallergenic tape. We will
also families to leave the temperature sensors on their child's body for 7
days. Families will repeat this process 4-6 weeks later as well.